FA Clinical Outcome Measures

Running, not enrolling

Conditions studied: Friedreich Ataxia, Neuro-Degenerative Disease

In brief

This multicenter natural history study aims to expand the network of clinical research centers in FA, and to provide a framework for facilitating therapeutic interventions. In addition, this study will lead to the development of valid yet sensitive clinical measures crucial to outcome assessment of patients with Friedreich's Ataxia. This study will support genetic modifier studies, biomarker studies, and frataxin protein level assessments by building a sample repository. This natural history study is no longer recruiting under this protocol NCT03090789 but remains actively recruiting under the harmonized study (UNIFAI) NCT06016946.

Key facts

Study ID

NCT03090789

Run by

Friedreich's Ataxia Research Alliance

People needed

2000

Starts

2001-01-01

Expected to finish

2030-01-01

Last updated by the study team

2024-10-04

Who can join

Age: 4 and older, up to 80. Sex: any. Healthy volunteers: accepted.

You may qualify if…

• Males or females age 4 to 80 years.
• Genetically confirmed diagnosis of FA (for carrier/control cheek swab and blood samples this is not required).
• Clinically confirmed diagnosis of FA, pending confirmatory genetic testing through a commercial or research laboratory (for carrier/control cheek swab and blood samples this is not required).
• Parental/guardian permission (informed consent) and if appropriate, child assent.

You may not qualify if…

• Signs or symptoms of severe cardiomyopathy (such as congestive heart failure)

Where it is running

• UCLA Ataxia Center — Los Angeles, California, United States
• University of Colorado — Denver, Colorado, United States
• University of Florida - Neurology — Gainesville, Florida, United States
• USF Ataxia Research Center — Tampa, Florida, United States
• Emory University Hospital - Neurology — Atlanta, Georgia, United States
• University of Iowa, Stead Family Children's Hospital — Iowa City, Iowa, United States
• Ohio State University - Neurology — Columbus, Ohio, United States
• Children's Hospital of Philadelphia - Neurology — Philadelphia, Pennsylvania, United States
• St. Jude Children's Research Hospital — Memphis, Tennessee, United States
• Murdoch Childrens Research Institute — Parkville, Victoria, Australia
• The Hospital for Sick Children — Toronto, Ontario, Canada
• CHUM - Hopital Notre-Dame — Montreal, Quebec, Canada
• All India Institute of Medical Sciences (Aiims) — New Delhi, India
• Auckland City Hospital — Auckland, New Zealand

Full record on ClinicalTrials.gov

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